Prognostic Implication of the United States Food and Drug Administration-defined BCG-unresponsive Disease
Prognostic Implication of the United States Food and Drug Administration-defined BCG-unresponsive Disease
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DOI：
10.1016/j.eururo.2018.09.028
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发表时间：
2019-01-01
期刊：
影响因子：
23.4
通讯作者：
Kamat, Ashish M.
中科院分区：
文献类型：
--
作者：
Li, Roger;Tabayoyong, William B.;Kamat, Ashish M.
卡介苗无反应性疾病类别是由专家应美国食品和药物管理局的要求制定的，是指一组患有复发性非肌层浸润性膀胱癌的患者，持续的卡介苗治疗不太可能为其带来益处。尽管很快被用于试验设计，但该定义中的许多细微差别缺乏验证。在这项研究中，我们通过比较这些患者的肿瘤学结果与仅诱导 BCG 后复发的患者，评估了 BCG 无反应指定（即诱导后复发加至少 1 个 BCG 维持疗程）的预后价值。我们确认，适当定义的、BCG 无反应的患者更有可能需要挽救性根治性膀胱切除术（54.5% vs 17.9%，p = 0.002）。此外，那些选择二线膀胱保留疗法的患者不太可能保持肿瘤复发（23% vs 69.2%，p = 0.003）。在多变量分析中，卡介苗无反应疾病独立预测较差的高级无复发生存期（风险比 [HR]：6.25，95% 置信区间 [CI]：2.2716.67；p < 0.001）和无膀胱切除生存期（HR：3.85，95% CI：1.4910.0；p = 0.006）。我们的数据证实了 BCG 无反应定义的预后意义，即诱导和一个疗程的 BCG 维持后重度疾病复发，并支持其在 BCG 后肿瘤复发患者的咨询和风险分层中的应用。膀胱切除术或临床试验入组 (C) 2018 年欧洲泌尿外科协会。由 Elsevier B.V. 出版。保留所有权利。
The category BCG-unresponsive disease, formulated by experts at the request of the United States Food and Drug Administration, denotes a group of patients with recurrent non-muscle-invasive bladder cancer for whom continued BCG treatment is unlikely to provide benefit. Although quickly adopted for trial design, many of the nuances within the definition lack validation. In this study, we evaluated the prognostic value of BCG unresponsive designation (i.e. recurrence after induction plus at least 1 maintenance course of BCG) by comparing the oncologic outcomes of these patients with those recurring after induction BCG alone. We confirm that appropriately defined, BCG-unresponsive patients are more likely to require salvage radical cystectomy (54.5% vs 17.9%, p = 0.002). Moeover, those opting for second-line bladder-sparing therapies are less likely to remain free of tumor recurrence (23% vs 69.2%, p = 0.003). On multivariate analysis, BCG-unresponsive disease independently predicts inferior high-grade recurrence-free survival (hazard ratio [HR]: 6.25, 95% confidence interval [CI]: 2.2716.67; p < 0.001) and cystectomy-free survival (HR: 3.85, 95% CI: 1.4910.0; p = 0.006). Our data confirm the prognostic implication of the BCG unresponsive definition i.e. recurrence of high grade disease after induction and one course of maintenance BCG, and support its use in counseling and risk stratification of patients with tumor recurrence after BCGPatient summary: Patients who have BCG-unresponsive disease, that is, high-grade non-muscle-invasive bladder cancer recurring after BCG induction and maintenance, have a low likelihood to respond to further BCG treatment and should consider radical cystectomy or clinical trial enrollment (C) 2018 European Association of Urology. Published by Elsevier B.V. All rights reserved.